Rondinone Cristina M
Department Metabolic Diseases, Hoffmann-La Roche, Nutley, NJ 07110, USA.
Biotechniques. 2006 Apr;Suppl:31-6. doi: 10.2144/000112163.
Over the past years RNA interference (RNAi) has exploded as a new approach to manipulate gene expression in mammalian systems. More recently, RNAi has acquired interest as a potential therapeutic strategy. This review focuses on the potential therapeutic use of RNAi for metabolic diseases, the current understanding of RNAi biology, and how RNAi has been utilized to study the role of different genes in the pathogenesis of diabetes and obesity. Also reviewed are the in vivo proof-of-principle experiments that provide the preclinical justification for the development of RNAi-based therapeutics for diabetes and the key challenges that currently limit its application in the clinical setting.
在过去几年中，RNA干扰（RNAi）已成为一种在哺乳动物系统中操纵基因表达的新方法。最近，RNAi作为一种潜在的治疗策略受到了关注。本综述重点关注RNAi在代谢性疾病中的潜在治疗用途、对RNAi生物学的当前理解，以及RNAi如何被用于研究不同基因在糖尿病和肥胖发病机制中的作用。还综述了体内原理验证实验，这些实验为开发基于RNAi的糖尿病治疗药物提供了临床前依据，以及目前限制其在临床应用的关键挑战。
